SAN DIEGO, March 11, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for conversion of the existing U.S. accelerated approval of FILSPARI® (sparsentan) in IgA nephropathy (IgAN) to full approval.

圣地亚哥，2024年3月11日（环球通讯社）--Travere Therapeutics，Inc.（纳斯达克：TVTX）今天宣布向美国食品和药物管理局（FDA）提交补充新药申请（sNDA），以将现有的美国加速批准用于IgA肾病（IgAN）的FILSPARI®（sparsentan）转化为完全批准。

In February 2023, the FDA granted accelerated approval to FILSPARI as the first and only non-immunosuppressive treatment targeting glomerular injury in the kidney to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression. The sNDA is based on 2-year confirmatory results from the Phase 3 PROTECT Study, the only head-to-head study in IgAN versus an active comparator.

2023年2月，FDA加速批准FILSPARI作为第一种也是唯一一种针对肾脏肾小球损伤的非免疫抑制治疗方法，以减少患有快速疾病进展风险的原发性IgAN成人的蛋白尿。sNDA基于3期PROTECT研究的2年验证结果，该研究是IgAN与主动比较者之间唯一的头对头研究。

“Since being introduced under accelerated approval, FILSPARI has positively impacted the lives of many people living with IgAN. The submission of the sNDA is an important step toward potentially gaining full approval in IgAN in support of reaching more people living with this devastating rare kidney disease,” said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics.

“自从在加速批准下引入FILSPARI以来，它对许多IgAN患者的生活产生了积极影响。sNDA的提交是朝着获得IgAN完全批准的方向迈出的重要一步，以支持更多患有这种毁灭性罕见肾脏疾病的患者，”Eric Dube博士说。，Travere Therapeutics总裁兼首席执行官。

“FILSPARI is at the forefront of emerging new treatment options providing hope for a delay in kidney transplant or dialysis. The results from the pivotal Phase 3 PROTECT Study show that by directly targeting glomerular injury in the kidney with FILSPARI, patients can achieve sustained proteinuria reduction and long-term kidney function preservation.

“FILSPARI处于新兴治疗方案的前沿，为延迟肾脏移植或透析提供了希望。关键的3期PROTECT研究的结果表明，通过使用FILSPARI直接靶向肾脏中的肾小球损伤，患者可以实现持续的蛋白尿减少和长期肾功能保存。

We look forward to working with the FDA throughout the upcoming review process.”

我们期待着在即将到来的审查过程中与FDA合作。”

FILSPARI is a once-daily, oral medication that directly targets glomerular injury in the kidney by blocking two critical pathways of IgAN disease progression (endothelin-1 and angiotensin II). FILSPARI is also the first and only non-immunosuppressive therapy approved for the treatment of this rare kidney disease.

FILSPARI是一种每日一次的口服药物，通过阻断IgAN疾病进展的两个关键途径（内皮素-1和血管紧张素II）直接靶向肾脏中的肾小球损伤。FILSPARI也是第一个也是唯一一个被批准用于治疗这种罕见肾脏疾病的非免疫抑制疗法。

The sNDA submission is supported by results from the Phase 3 PROTECT Study that showed that FILSPARI demonstrated long-term kidney function preservation and achieved a significant reduction in proteinuria and a clinically meaningful difference in eGFR slope versus an active comparator.

sNDA的提交得到了第3阶段PROTECT研究结果的支持，该研究表明，FILSPARI表现出长期的肾功能保存，并显着降低了蛋白尿，并且与活性对照相比，eGFR斜率具有临床意义的差异。

The FDA has 60 days from the receipt of the application to determine whether to accept it for review. The Company expects to receive notice regarding the acceptance for review of the sNDA submission as well as the timeline for sNDA review from the FDA in the second quarter of 2024. In addition to the sNDA submission to the FDA, the Company and its European commercial partner CSL Vifor recently announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended approval of the conditional marketing authorization (CMA) for sparsentan for the treatment of IgA nephropathy (IgAN) in Europe.

FDA在收到申请后60天内决定是否接受审查。该公司预计将于2024年第二季度收到FDA关于接受sNDA提交审查的通知以及sNDA审查时间表。除了sNDA提交给FDA之外，该公司及其欧洲商业合作伙伴CSL Vifor最近宣布，欧洲药品管理局（EMA）人类使用药品委员会（CHMP）建议批准斯巴森坦在欧洲治疗IgA肾病（IgAN）的有条件上市授权（CMA）。

A decision by the European Commission is expected in the second quarter of 2024. If approved, sparsentan would receive a CMA in all member states of the European Union, as well as in Iceland, Liechtenstein, and Norway.

预计欧盟委员会将在2024年第二季度做出决定。如果获得批准，sparsentan将在欧盟所有成员国以及冰岛、列支敦士登和挪威获得CMA。

About IgA Nephropathy

关于IgA肾病

IgA nephropathy (IgAN), also called Berger's disease, is a rare progressive kidney disease characterized by the buildup of immunoglobulin A (IgA), a protein that helps the body fight infections, in the kidneys. The deposits of IgA cause a breakdown of the normal filtering mechanisms in the kidney, leading to blood in the urine (hematuria), protein in the urine (proteinuria) and a progressive loss of kidney function.

IgA肾病（IgAN），也称为伯杰病，是一种罕见的进行性肾脏疾病，其特征是免疫球蛋白a（IgA）在肾脏中积聚，免疫球蛋白a是一种帮助身体抵抗感染的蛋白质。IgA的沉积导致肾脏正常过滤机制的破坏，导致尿液中的血液（血尿），尿液中的蛋白质（蛋白尿）和肾功能的进行性丧失。

Other symptoms of IgAN may include swelling (edema) and high blood pressure.

IgAN的其他症状可能包括肿胀（水肿）和高血压。

IgAN is the most common type of primary glomerulonephritis worldwide and a leading cause of kidney failure due to glomerular disease. IgAN is estimated to affect up to 150,000 people in the U.S. and is one of the most common glomerular diseases in Europe and Japan.

IgAN是全球最常见的原发性肾小球肾炎类型，也是肾小球疾病导致肾衰竭的主要原因。据估计，IgAN在美国影响多达15万人，是欧洲和日本最常见的肾小球疾病之一。

About the PROTECT Study

关于PROTECT研究

The PROTECT Study is one of the largest interventional studies to date in IgA nephropathy (IgAN) and the only head-to-head trial in this rare kidney disease. It is a global, randomized, multicenter, double-blind, parallel-arm, active-controlled clinical trial evaluating the safety and efficacy of 400 mg of sparsentan, compared to 300 mg of irbesartan, in 404 patients ages 18 years and up with IgAN and persistent proteinuria despite receiving at least 50% of max label dose and maximally tolerated ACE or ARB therapy.

PROTECT研究是迄今为止IgA肾病（IgAN）中最大的介入研究之一，也是这种罕见肾脏疾病中唯一的头对头试验。这是一项全球性，随机，多中心，双盲，平行组，主动对照临床试验，评估了404名18岁及以上IgAN和持续性蛋白尿患者中400 mg斯巴森坦（与300 mg厄贝沙坦相比）的安全性和有效性，尽管接受了至少50%的最大标记剂量和最大耐受性ACE或ARB治疗。

In August 2021, the Company announced the PROTECT Study met its pre-specified interim primary efficacy endpoint with statistical significance. Based on the pre-specified, primary analyses set, after 36 weeks of treatment, patients receiving sparsentan achieved a mean reduction in proteinuria from baseline of 49.8%, compared to a mean reduction in proteinuria from baseline of 15.1% for irbesartan-treated patients (p<0.0001).

2021年8月，该公司宣布PROTECT研究符合其预先指定的中期主要疗效终点，具有统计学意义。根据预先指定的初步分析数据集，治疗36周后，接受斯巴森坦治疗的患者的蛋白尿平均从基线水平降低了49.8%，而厄贝沙坦治疗的患者的蛋白尿平均从基线水平降低了15.1%（p<0.0001）。

The study’s confirmatory secondary endpoint in the U.S. is estimated glomerular filtration rate (eGFR) total slope from day 1 to week 110 of treatment. The confirmatory secondary endpoint in the EU is eGFR chronic slope from week 6 to week 110 of treatment, following the initial acute effect of randomized treatment.

该研究在美国的证实性次要终点是估计治疗第1天至第110周的肾小球滤过率（eGFR）总斜率。在随机治疗的初始急性效应之后，欧盟的确诊次要终点是治疗第6周至第110周的eGFR慢性斜率。

Following the 110-week blinded treatment period, treatment with study medication was discontinued for 4 weeks -- at this time, the investigator resumed standard of care treatment. In September 2023, the Company announced topline two-year confirmatory secondary endpoint results from the PROTECT Study of sparsentan in IgAN.

在110周的盲法治疗期后，研究药物的治疗中断了4周-此时，研究者恢复了护理标准治疗。2023年9月，该公司宣布了IgAN中sparsentan PROTECT研究的topline两年验证性次要终点结果。

Sparsentan demonstrated long-term kidney function preservation and achieved a clinically meaningful difference in eGFR total and chronic slope versus irbesartan, narrowly missing statistical significance in eG.

Sparsentan表现出长期的肾功能保存，并且与厄贝沙坦相比，eGFR总量和慢性斜率具有临床意义的差异，在eG中几乎没有统计学意义。

About Travere Therapeutics

关于Travere Therapeutics

At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies.

在Travere Therapeutics，我们的生命是罕见的。我们是一家生物制药公司，每天都会齐聚一堂，帮助各种背景的患者、家属和护理人员应对罕见疾病。在这条道路上，我们知道迫切需要治疗选择，这就是为什么我们的全球团队与罕见病社区合作，以确定、开发和提供改变生命的疗法。

In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow.

在追求这一使命的过程中，我们不断寻求了解罕见患者的不同观点，并勇敢地开辟新的道路，改变他们的生活，为他们的今天和明天带来希望。

FILSPARI® (sparsentan) U.S. Indication

菲尔斯巴利® （稀疏）美国指示

FILSPARI is an endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g.

FILSPARI是一种内皮素和血管紧张素II受体拮抗剂，可减少患有原发性免疫球蛋白A肾病（IgAN）的成年人的蛋白尿，其疾病进展迅速，通常UPCR≥1.5 g/g。

This indication is granted under accelerated approval based on reduction in proteinuria. It has not been established whether FILSPARI slows kidney function decline in patients with IgAN. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial.

该适应症是在蛋白尿减少的基础上加速批准的。尚未确定FILSPARI是否能减缓IgAN患者的肾功能下降。是否继续批准该适应症可能取决于验证性临床试验中临床益处的验证和描述。